Академический Документы
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Medicines in Development
Biologics
presented by america’s biopharmaceutical research companies
338
Application
Submitted
Phase III
Phase II
America’s biopharmaceutical research • 58 treatments for cardiovascular
Phase I companies are developing 907 biologics diseases, such as congestive heart
250 targeting more than 100 diseases. Biologics failure and stroke.
are developed through biological processes • Other diseases include diabetes,
using living cells or organisms. digestive disorders, genetic disorders,
This report lists biologics in human neurologic and respiratory disorders.
clinical trials or under review by the Definitions for selected product categories
U.S. Food and Drug Administration and diseases can be found on page 86.
(FDA). A link to the sponsor company’s
web site provides more detailed infor- For information on the history and
mation on the science and technologies future of biologics research, the science
behind each potential product. The 907 behind the medicines, and a discussion
medicines and vaccines in development of issues critical to continued discovery
include: and development of these cutting-edge
medicines, please see Biologic Medicines
• 338 cancer therapeutics that target in Development 2013—Overview.
93
several different types of solid tumors,
81
leukemia and lymphoma. Monoclonal The medicines in this report reflect the
69
antibodies account for 170 of the 338 new ways America’s biopharmaceuti-
products in development. cal research companies are attacking
46 disease through biotechnology. The 907
• 176 candidates in development for an medicines and vaccines in development
30 array of infectious diseases, including promise to push the frontiers of science
134 vaccines. and bring new treatments to patients for
• 71 medicines for autoimmune diseases, our more challenging diseases.
such as lupus, multiple sclerosis and
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Biologic Medicines in Development
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase*
Category
*For more information about a specific medicine or company in the report, please click on the provided link.
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
Ilaris® Novartis Pharmaceuticals mAb systemic juvenile idiopathic arthritis Phase III
canakinumab East Hanover, NJ (see also cardiovascular, diabetes, novartis.com
(Orphan Drug) musculoskeletal)
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
Autoimmune Disorders
Product Name Sponsor Product Indication Development Phase
Category
Blood Disorders
Product Name Sponsor Product Indication Development Phase
Category
ART123 Asahi Kasei Pharma America recombinant disseminated intravascular Phase III
(recombinant human Waltham, MA protein coagulation akpamerica.com
thrombomodulin alpha)
ATryn® GTC Biotherapeutics recombinant heparin resistance in patients under- Phase III
antithrombin alfa Framingham, MA protein going CABG surgery gtc-bio.com
(Orphan Drug)
Blood Disorders
Product Name Sponsor Product Indication Development Phase
Category
Blood Disorders
Product Name Sponsor Product Indication Development Phase
Category
NiCord® Gamida Cell stem cell sickle cell anemia Phase I/II
umbilical cord blood Jerusalem, Israel therapy (see also cancer) gamida-cell.com
stem cell therapy
NN1841 Novo Nordisk recombinant congenital factor XIII deficiency application submitted
(rFXIII) Princeton, NJ protein novonordisk.com
Blood Disorders
Product Name Sponsor Product Indication Development Phase
Category
ABIO-0501 Abiogen Pharma cell therapy chronic myeloid leukemia (CML) Phase II
(TALL-104) Pisa, Italy abiogen.it
Actimab-A Actinium Pharmaceuticals mAb acute myeloid leukemia (AML) Phase I/II
(M195 mAb) New York, NY actiniumpharmaceuti
cals.com
ALT-801 Altor BioScience recombinant AML, bladder cancer, malignant Phase I/II
Miramar, FL fusion melanoma, multiple myeloma altorbioscience.com
protein
ASC-101 America Stem Cell stem cell myeloablation associated with Phase I/II
(Orphan Drug) Carlsbad, CA therapy hematological malignancies americastemcell.com
autologous stem cell California Stem Cell stem cell malignant melanoma Phase II
therapy Irvine, CA therapy californiastemcell.com
breast cancer vaccine MabVax Therapeutics mAb vaccine breast cancer Phase I
San Diego, CA mabvax.com
calaspargase pegol Sigma-Tau Pharmaceuticals pegylated ALL in adolescents and children Phase III
Gaithersburg, MD enzyme sigmatau.com
cancer vaccine EMD Millipore dendritic cell malignant melanoma Phase I completed
Billerica, MA vaccine millipore.com
encapsulated cell therapy Metromedia Bio-Science cell colorectal cancer, pancreatic Phase II
New York, NY therapy cancer, prostate cancer rogosin.org
Rogosin Institute
New York, NY
folate binding protein-E39 Galena Biopharma peptide endometrial cancer, ovarian cancer Phase I/II
(FBP) Lake Oswego, OR vaccine galenabiopharma.com
mogamulizimab Kyowa Hakko Kirin Pharma mAb cutaneous T-cell lymphoma Phase III
(Orphan Drug) Princeton, NJ kyowa-kirin-pharma.com
------------------------------------------------ ---------------------------------
adult T-cell lymphoma, Phase II
T-cell leukemia kyowa-kirin-pharma.com
Multikine® CEL-SCI growth factor, head and neck cancer Phase III
leukocyte Vienna, VA interferon, cel-sci.com
interleukin cancer interleukin
(Orphan Drug)
nivolumab Bristol-Myers Squibb mAb melanoma, NSCLC, renal cell Phase III
(anti-PD1) Princeton, NJ carcinoma bms.com
----------------------------------------------- ---------------------------------
solid tumors Phase I/II
bms.com
obinutuzumab Biogen Idec mAb CLL, diffuse large B-cell lymphoma, Phase III
(GA101) Weston, MA non-Hodgkin lymphoma biogenidec.com
Genentech gene.com
South San Francisco, CA
ovarian cancer vaccine MabVax Therapeutics mAb vaccine ovarian cancer Phase II
San Diego, CA mabvax.com
polyclonal antibody Cancer Advances polyclonal gastric cancer, pancreatic cancer Phase III
stimulator Durham, NC antibody (see also digestive) canceradvances.com
(Orphan Drug) vaccine ------------------------------------------------ --------------------------------
colorectal cancer Phase II
canceradvances.com
ramucirumab Eli Lilly mAb breast cancer, colorectal cancer, Phase III
(LY3009806) Indianapolis, IN gastric cancer lilly.com
ImClone Systems
Princeton, NJ
SNS01-T Senesco Technologies gene diffuse large B-cell lymphoma, Phase I/II
Bridgewater, NJ therapy; RNA mantle cell lymphoma, senesco.com
interference multiple myeloma
Zevalin® Spectrum Pharmaceuticals mAb diffuse large B-cell lymphoma Phase III
ibritumomab tiuxetan Henderson, NV sppirx.com
------------------------------------------------ ---------------------------------
non-Hodgkin lymphoma Phase II
sppirx.com
Cardiovascular Disease
Product Name Sponsor Product Indication Development Phase
Category
adipose stem cell therapy Cytori Therapeutics stem cell myocardial ischemia Phase I/II
San Diego, CA therapy cytori.com
adult autologous stem cell Baxter International stem cell myocardial ischemia Phase III
therapy Deerfield, IL therapy baxter.com
------------------------------------------------ ---------------------------------
critical limb ischemia Phase I/II completed
baxter.com
adult mesenchymal stem BioCardia stem cell ischemic heart disorders Phase I/II
cell therapy San Carlos, CA therapy biocardia.com
allogeneic stem Stemedica Cell Technologies stem cell ischemic stroke Phase I/II
cell therapy San Diego, CA therapy stemedica.com
Cardiovascular Disease
Product Name Sponsor Product Indication Development Phase
Category
combination autologous TCA Cellular Therapy stem cell coronary ischemia, Phase II
stem cell therapy Covington, LA therapy ischemic heart disorders tcacellulartherapy.com
fibroblast growth CardioVascular BioTherapeutics fibroblast severe coronary heart disease Phase II
factor-1 Las Vegas, NV growth factor (see also skin) cvbt.com
Cardiovascular Disease
Product Name Sponsor Product Indication Development Phase
Category
ischemic tolerant Stemedica Cell Technologies stem cell acute myocardial infarction Phase II
allogeneic mesenchymal San Diego, CA therapy stemedica.com
stem cell therapy
ixmyelocel-T Aastrom Biosciences stem cell critical limb ischemia Phase III
(Orphan Drug) Ann Arbor, MI therapy (Fast Track) aastrom.com
------------------------------------------------ ---------------------------------
dilated cardiomyopathy Phase II
aastrom.com
MultiGeneAngio MultiGene Vascular Systems cell peripheral arterial disorders Phase I/II
Nesher, Israel therapy mgvs.co.il
Cardiovascular Disease
Product Name Sponsor Product Indication Development Phase
Category
SDF-1 Juventas Therapeutics gene therapy critical limb ischemia, heart failure Phase II
(stromal cell-derived Cleveland, OH juventasinc.com
factor-1)
Cardiovascular Disease
Product Name Sponsor Product Indication Development Phase
Category
Diabetes
Product Name Sponsor Product Indication Development Phase
Category
Diabetes
Product Name Sponsor Product Indication Development Phase
Category
insulin glargine Sanofi US recombinant type 1 diabetes, type 2 diabetes Phase III
(rDNA origin) Bridgewater, NJ insulin sanofi.com
(new formulation)
LY2605541 Boehringer Ingelheim insulin type 1 diabetes, type 2 diabetes Phase III
(novel basal insulin/ Pharmaceuticals hormone boehringer-ingelheim.
insulin peglispro) Ridgefield, CT com
Eli Lilly lilly.com
Indianapolis, IN
LY2963016 Boehringer Ingelheim insulin type 1 diabetes, type 2 diabetes Phase III
(new insulin glargine Pharmaceuticals hormone boehringer-ingelheim.
product) Ridgefield, CT com
Eli Lilly lilly.com
Indianapolis, IN
Diabetes
Product Name Sponsor Product Indication Development Phase
Category
Digestive Disorders
Product Name Sponsor Product Indication Development Phase
Category
Digestive Disorders
Product Name Sponsor Product Indication Development Phase
Category
Prochymal® Osiris Therapeutics stem cell Crohn’s disease (Fast Track), Phase III
remestemcel-L Columbia, MD therapy gastrointestinal injury from osiris.com
radiation exposure
(see also cardiovascular, diabetes,
respiratory, transplantation)
Digestive Disorders
Product Name Sponsor Product Indication Development Phase
Category
QAX576 Novartis Pharmaceuticals mAb rectal fistula in patients with Crohn’s Phase II
East Hanover, NJ disease novartis.com
(see also respiratory)
vedolizumab Takeda Pharmaceuticals mAb Crohn’s disease, ulcerative colitis Phase III
(MLN0002) Deerfield, IL takeda.com
Eye Conditions
Product Name Sponsor Product Indication Development Phase
Category
AVA-101 Avalanche Biotechnologies gene therapy age-related macular degeneration Phase I/II
San Francisco, CA avalanchebiotech.com
Eye Conditions
Product Name Sponsor Product Indication Development Phase
Category
human retinal Advanced Cell Technology stem cell dry age-related macular Phase I/II
pigment epithelial Santa Monica, CA therapy degeneration, Stargardt’s advancedcell.com
cell therapy macular degeneration
(Orphan Drug)
Eye Conditions
Product Name Sponsor Product Indication Development Phase
Category
Eye Conditions
Product Name Sponsor Product Indication Development Phase
Category
Genetic Disorders
Product Name Sponsor Product Indication Development Phase
Category
Genetic Disorders
Product Name Sponsor Product Indication Development Phase
Category
KRN-23 Kyowa Hakko Kirin Pharma mAb X-linked dominant Phase I/II
Princeton, NJ hypophosphatemic rickets kyowa-kirin-pharma.com
Genetic Disorders
Product Name Sponsor Product Indication Development Phase
Category
leber congenital Applied Genetic Technologies gene leber congenital amaurosis Phase I/II
amaurosis gene Alachua, FL therapy agtc.com
therapy
(Orphan Drug)
WAS gene therapy Children’s Hospital Boston gene therapy Wiskott-Aldrich syndrome Phase I/II
Boston, MA genethon.fr
Genethon
Évry, France
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
acellular pertussis combo Novartis Vaccines vaccine diphtheria, pertussis, tetanus Phase I
vaccine Cambridge, MA novartisvaccines.com
ADVAX Aaron Diamond AIDS DNA HIV infection prevention Phase I completed
(DNA vaccine) Research Center vaccine adarc.org
New York, NY iavi.org
International AIDS Vaccine ------------------------------------------------ ---------------------------------
Initiative HIV infection prevention Phase I completed
New York, NY (new delivery system) adarc.org
Ichor Medical Systems iavi.org
San Diego, CA
Agriflu® Novartis Vaccines vaccine influenza virus infections (pediatric) Phase III
influenza virus vaccine Cambridge, MA ention) novartisvaccines.com
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
dengue vaccine Sanofi Pasteur vaccine mild to severe dengue fever Phase III
Swiftwater, PA sanofipasteur.us
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
Ebola virus vaccine GenPhar vaccine Ebola virus infections in clinical trials
Mt. Pleasant, SC genphar.com
Fluzone® QIV IM Sanofi Pasteur vaccine seasonal influenza virus infections application submitted
quadrivalent influenza Swiftwater, PA sanofipasteur.us
virus vaccine
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
HBV-002 Hawaii Biotech recombinant prevention of West Nile virus Phase I completed
Aiea, Hawaii vaccine infections hibiotech.com
Hexaxim™ Sanofi Pasteur vaccine diphtheria, tetanus, pertussis, hepa- application submitted
DTP-HepB-polio-Hib Swiftwater, PA titis B, poliomyelitis, sanofipasteur.us
vaccine Haemophilus influenzae type b
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
HIV vaccine PaxVax oral vaccine HIV infection prevention in clinical trials
San Diego, CA paxvax.com
HIV-1 gene therapy Takara Bio gene therapy HIV-1 infection Phase I
(MazF) Shiga, Japan takara-bio.com
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
HSV-2 Tx Genocea Biosciences T-cell vaccine treatment of herpes simplex virus 2 Phase I/II
Cambridge, MA infections genocea.com
influenza A virus H1N1 Medicago VLP vaccine influenza A virus H1N1 subtype Phase I
vaccine Quebec, Canada medicago.com
influenza A virus H1N1 Inovio Pharmaceuticals DNA vaccine influenza A virus H1N1 subtype Phase I
vaccine (FVH-1) Blue Bell, PA prevention inovio.com
influenza A virus H1N1 Antigen Express peptide influenza A virus H1N1 infections Phase I
vaccine (li-key hybrid Worcester, MA vaccine antigenexpress.com
peptide vaccine)
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
influenza A virus H5N1 Nanotherapeutics vaccine prevention of influenza A virus H5N1 Phase I
intranasal vaccine Alachua, FL subtype nanotherapeutics.com
influenza A virus H5N1 iBio vaccine influenza A virus H5N1 subtype Phase I
vaccine Newark, DE ibioinc.com
influenza A virus H5N1 Inovio Pharmaceuticals vaccine prevention of influenza A virus H5N1 Phase I
vaccine Blue Bell, PA subtype inovio.com
influenza A virus H5N1 Medicago VLP vaccine influenza A virus H5N1 subtype Phase I
vaccine Quebec, Canada medicago.com
influenza A virus H5N1 Novartis Vaccines vaccine prevention of influenza A virus Phase II
vaccine Cambridge, MA H5N1 subtype novartisvaccines.com
influenza A virus H5N1 Antigen Express peptide influenza A virus H5N1 subtype Phase I
vaccine Worcester, MA vaccine antigenexpress.com
(li-key hybrid peptide
vaccine)
influenza A virus H9N2 Baxter International vaccine influenza A virus H9N2 subtype Phase I/II
vaccine Deerfield, IL baxter.com
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
Lyme disease vaccine Baxter Healthcare recombinant prevention of Lyme disease Phase I/II
Deerfield, IL vaccine baxter.com
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
MMR vaccine live GlaxoSmithKline vaccine prevention of measles, mumps, Phase III
(GSK209762) Rsch. Triangle Park, NC rubella gsk.com
MnB rLP2086 Pfizer recombinant adolescent and young adult Phase III
(PF-05212366) New York, NY vaccine meningitis B pfizer.com
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
MVA-BN® Bavarian Nordic vaccine HIV-1 infection prevention and Phase I/II
HIV multiantigen vaccine Washington, DC treatment bavarian-nordic.com
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
Stealth Vector® Enzo Therapeutics antisense HIV-1 infection treatment Phase I/II
HGTV-43™ New York, NY enzo.com
antisense gene medicine
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
VaxiGrip® Sanofi Pasteur vaccine seasonal influenza virus infections Phase III
quadrivalent inactivated Swiftwater, PA sanofipasteur.us
influenza vaccine
Infectious Diseases
Product Name Sponsor Product Indication Development Phase
Category
Musculoskeletal Disorders/Arthritis
Product Name Sponsor Product Indication Development Phase
Category
Musculoskeletal Disorders/Arthritis
Product Name Sponsor Product Indication Development Phase
Category
FGF-18 EMD Serono growth factor acute cartilage injury of the knee Phase II
(sprifermin) Rockland, MA emdserono.com
Musculoskeletal Disorders/Arthritis
Product Name Sponsor Product Indication Development Phase
Category
teriparatide Eli Lilly recombinant healing of fractures in patients with Phase III
(recombinant PTH 1-34) Indianapolis, IN hormone osteoporosis lilly.com
Neurologic Disorders
Product Name Sponsor Product Indication Development Phase
Category
Neurologic Disorders
Product Name Sponsor Product Indication Development Phase
Category
GDNF-producing adult BrainStorm Cell Therapeutics stem cell amyotrophic lateral sclerosis Phase I/II
stem cell therapy New York, NY therapy (ALS) brainstorm-cell.com
(Orphan Drug)
Neurologic Disorders
Product Name Sponsor Product Indication Development Phase
Category
HuCNS-SC® StemCells stem cell thoracic spinal cord injury Phase I/II
adult neural stem Newark, CA therapy (see also eye, genetic) stemcellsinc.com
cell therapy
(intraosseous)
neurological disorders TCA Cellular Therapy stem cell ALS, spinal cord injuries Phase I
stem cell therapy Covington, LA therapy tcacellulartherapy.com
Neurologic Disorders
Product Name Sponsor Product Indication Development Phase
Category
Respiratory Disorders
Product Name Sponsor Product Indication Development Phase
Category
ALT-836 Altor BioScience mAb acute lung injury, adult respiratory Phase II
Miramar, FL distress syndrome altorbioscience.com
Genentech (see also cancer) gene.com
South San Francisco, CA
Respiratory Disorders
Product Name Sponsor Product Indication Development Phase
Category
grass pollen extract Stallergenes immuno- seasonal allergic rhinitis Phase III
Cedex, France therapy stallergenes.com
Respiratory Disorders
Product Name Sponsor Product Indication Development Phase
Category
Pollinex® Quattro Allergy Therapeutics vaccine grass pollen hypersensitivity, Phase III
injectable MPL allergy West Sussex, United Kingdom ragweed pollen hypersensitivity allergytherapeutics.com
vaccine ----------------------------------------------- ---------------------------------
tree pollen hypersensitivity Phase II
allergytherapeutics.com
ragweed allergy vaccine Greer Laboratories vaccine seasonal allergic rhinitis Phase III
(sublingual) Lenoir, NC greerlabs.com
Respiratory Disorders
Product Name Sponsor Product Indication Development Phase
Category
Skin Diseases
medicines for skin cancer are listed under Cancer
Skin Diseases
medicines for skin cancer are listed under Cancer
Product Name Sponsor Product Indication Development Phase
Category
Skin Diseases
medicines for skin cancer are listed under Cancer
Product Name Sponsor Product Indication Development Phase
Category
Transplantation
Product Name Sponsor Product Indication Development Phase
Category
mesenchymal precursor Mesoblast stem cell for bone marrow regeneration Phase III
cell (MPC) product New York, NY therapy in patients undergoing bone mesoblast.com
(Orphan Drug) marrow transplantation
(see also cardiovascular, diabetes,
musculoskeletal)
Transplantation
Product Name Sponsor Product Indication Development Phase
Category
Other
Product Name Sponsor Product Indication Development Phase
Category
Ampligen® Hemispherx Biopharma RNA product chronic fatigue syndrome application submitted
rintatolimid Philadelphia, PA hemispherx.net
(Orphan Drug)
Other
Product Name Sponsor Product Indication Development Phase
Category
cat allergy vaccine Circassia vaccine hypersensitivity in patients with Phase III
Oxford, United Kingdom rhinoconjunctivitis caused by cat circassia.co.uk
allergy
Other
Product Name Sponsor Product Indication Development Phase
Category
hMaxi-K Ion Channel Innovations gene therapy overactive bladder Phase I completed
gene therapy New York, NY ionchannelinnovations.
com
JRC-LAMP-vax Immunomic Therapeutics DNA vaccine Japanese red cedar pollen-induced Phase I
Lancaster, PA rhinoconjunctivitis immunomix.com
kidney disease medicine Eli Lilly biologic chronic kidney disease Phase I
Indianapolis, IN lilly.com
liver cell therapy Cytonet cell urea cycle disorders in children Phase II
(Orphan Drug) Weinheim, Germany therapy and infants cytonet.de
Other
Product Name Sponsor Product Indication Development Phase
Category
QPI-1002 Quark Pharmaceuticals RNA prevention of renal failure in patients Phase I/II
(I5Np) Fremont, CA interference undergoing cardiovascular surgery quarkpharma.com
(see also transplantation)
Other
Product Name Sponsor Product Indication Development Phase
Category
adjuvant—Substance or drug that aids an- erythropoietin—Hormone that stimulates red can be reactivated to produce painful sores of
other substance in its action. blood cell production in the bone marrow. the anus or genitals.
angioedema—Swelling of the mucous mem- Fast Track—A process designed to facilitate human papillomavirus—Viral agent of warts,
branes, tissues beneath the skin, or an internal the development and expedite the review believed to be contagious and usually harm-
organ due to an allergic reaction. of drugs to treat serious diseases and fill an less, but it can lead to cervical cancer.
unmet medical need. The status is assigned
ankylosing spondylitis—An inflammatory interferon—A glycoprotein naturally produced
by the U.S. Food and Drug Administration.
disorder of unknown cause that primarily by cells that interferes with the ability of a virus
The purpose is to get important new drugs
affects the spine. The vertebrae may fuse to reproduce after it invades the body.
to the patient earlier. Fast Track addresses a
together and form a rigid back that is impos-
broad range of serious diseases. Generally, interleukin—An endogenous substance that
sible to bend.
determining factors include whether the drug stimulates the production of different types of
antisense—An antisense drug is the mirror or will have an impact on such factors as survival, white blood cells or leukocytes.
complementary image of a small segment of day-to-day functioning, or the likelihood that
the disease, if left untreated, will progress from ischemia—Insufficient supply of blood to an
messenger RNA (mRNA), the substance that
a less severe condition to a more serious one. organ or tissue, which can cause organ dam-
carries instructions (“sense”) from the genes to
Filling an unmet medical need is defined as pro- age such as an ischemic stroke.
the cell’s protein-making machinery. The anti-
sense drug readily binds to the mRNA strand, viding a therapy where none exists or providing lymphoma—Cancers in which the cells of lym-
keeping it from transmitting its instructions to a therapy which may be potentially superior phoid tissue, found mainly in the lymph nodes
the cell, and thus inhibiting the production of an to existing therapy. Once a drug receives Fast and spleen, multiply unchecked. Lymphomas
unwanted protein. Track designation, early and frequent commu- fall into two categories: One is called Hodgkin
nication between the FDA and a drug company disease, characterized by a particular kind
application submitted—An application for is encouraged throughout the entire drug de- of abnormal cell. All others are called non-
marketing has been submitted by the company velopment and review process. The frequency Hodgkin lymphomas, which vary in their
to the Food and Drug Administration (FDA). of communication assures that questions and malignancy according to the nature and activity
biotechnology—The collection of processes issues are resolved quickly, often leading to of the abnormal cells.
that involve the use of biological systems. earlier drug approval and access by patients.
mAb (monoclonal antibodies)—Large protein
For some of the industries, these processes gene therapy—Therapy at the intracellular molecules produced by white blood cells that
involve the use of genetically engineered level to replace or inactivate the effects of seek out and destroy harmful foreign sub-
organisms. For the purpose of this report, only disease-causing genes or to augment normal stances.
those products that involve recombinant DNA, gene functions to overcome illness.
monoclonal antibody/hybridoma, continuous macular degeneration—A progressive disor-
cell lines, cellular therapy, gene therapy and glioblastoma—The most common primary der that affects the central part of the retina,
vaccines technology are included. brain tumor and one of the most aggressive causing gradual loss of vision. It is a painless
forms of brain cancer, primarily affecting adults condition, usually affects both eyes and is com-
Clostridium difficile—A bacterium that over the age of 50. mon in the elderly.
produces an irritating toxin that causes a form
of colitis characterized by profuse, watery diar- growth factors—Factors responsible for metastases/metastatic—Cancers that have
rhea with cramps and low-grade fever. regulating cell proliferation (rapid and repeated spread from the primary or original cancer site.
reproduction), function and differentiation. Cancer cells can break away from a tumor and
colony stimulating factor (CSF)—Protein
growth hormone—Pituitary hormone that travel to other areas of the body through the
responsible for controlling the production of
stimulates the growth of long bones in prepu- bloodstream or the lymph system. They are of-
white blood cells.
bertal children. ten more difficult to treat then primary tumors.
Crohn’s disease—A subacute chronic gastro-
hemophilia A and B—Hemophilia A, the “clas- multiple myeloma—A malignant condition char-
intestinal disorder, involving the small intestine,
sic” hemophilia, is a genetic bleeding disorder acterized by uncontrolled proliferation of plasma
characterized by patchy deep ulcers that may
due to deficiency of the coagulation factor cells (a class of white blood cells) in bone
cause fistulas and a narrowing and thickening
VIII. Hemophilia B, or “Christmas” disease, is marrow. Symptoms include pain and destruction
of the bowel.
caused by deficiency of coagulation factor IX. of bone tissue, numbness and paralysis, kidney
cytomegalovirus (CMV)—A DNA virus related damage, anemia, and frequent infections.
to the herpes virus, affecting mostly neonatal hepatitis—Inflammation of the liver with accom-
panying liver cell damage or death, caused most myelodysplastic syndromes (MDS)—Blood
infants and immunocompromised individuals.
often by viral infection, e.g., hepatitis B, and C. disorders that ultimately are fatal. Patients
CMV can occur without symptoms or result in
usually succumb to infections or bleeding. The
mild flu-like symptoms.
herpes simplex virus—A strain of herpes term “preleukemia” has been used to describe
virus that may lie dormant in nerve tissue and these disorders.
myeloid—Pertaining to, derived from, or mani- Phase I—Researchers test the drug in a small systemic lupus erythematosus (SLE)—The
festing certain features of the bone marrow. In group of people, usually between 20 and 80 most serious form of lupus, a chronic autoim-
some cases, myeloid also pertains to certain healthy adult volunteers, to evaluate its initial mune disorder causing inflammation and
types of non-lymphocyte white blood cells damage to multiple organs.
safety and tolerability profile, determine a
found in the bone marrow.
safe dosage range, and identify potential side thrombocytopenia—A reduction in the
myocardial infarction—Damage to the effects. number of platelet cells in the blood, which
heart muscle caused by stoppage or impair- causes a tendency to bleed, especially from
ment of blood flow to the heart, also known Phase II—The drug is given to volunteer the smaller blood vessels.
as heart attack. patients, usually between 100 and 300, to see ulcerative colitis—A chronic inflammation and
neuroblastoma—A tumor of the adrenal if it is effective, identify an optimal dose, and to ulceration of the lining of the colon and rectum.
glands or sympathetic nervous system (the further evaluate its short-term safety. It causes bloody diarrhea and mainly involves
part of the nervous system responsible for the left colon.
certain automatic body functions, such as the Phase III—The drug is given to a larger, more
diverse patient population, often involving be- uveitis—Inflammation of the uvea, the middle
“fight or flight response”).
layer of the eye.
tween 1,000 and 3,000 patients (but sometime
neutropenia—An abnormally low neutrophil
count (certain white blood cells). many more thousands), to generate statistically
significant evidence to confirm its safety and
Orphan Drug—A drug to treat a disease that
effectiveness. They are the longest studies,
has a patient population of 200,000 or less
in the United States, or a disease that has a and usually take place in multiple sites around
patient population of more than 200,000 and the world.
a development cost that will not be recovered pulmonary fibrosis—Scarring and thickening
from sales in the United States. of lung tissue.
peripheral vascular disorder—The obstruc- respiratory syncytial virus (RSV) disease—
tion of blood supply to the extremities, particu- One of the most important causes of lower
larly the legs, caused by atherosclerosis. respiratory tract disease in children, account-
Phase 0—First-in-human trials conducted ing for more than 90 percent of cases of
in accordance with FDA’s 2006 guidance on bronchiolitis.
exploratory Investigational New Drug (IND) restenosis—A condition where an artery plugs
studies designed to speed up development of up again following treatment to open it up.
promising drugs by establishing very early on
whether the agent behaves in human subjects sepsis—A condition associated with a serious
as was anticipated from preclinical studies. bacterial infection of the blood.
The content of this report has been obtained through public, government and industry sources, and the Adis “R&D Insight” database based on the
latest information. Report current as of February 7, 2013. The information in this report may not be comprehensive. For more specific information
about a particular product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is
available on PhRMA’s web site.
A publication of PhRMA’s Communications & Public Affairs Department. (202) 835-3460
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